patient advocacy

For me, getting diagnosed with late-onset Pompe disease was a relief. Although there’s no cure, treatment is available to help slow disease progression. After starting treatment, I wondered what my life would look like. Would I have to stay home? How long would I be able…

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…

Health advocacy can help improve the quality and accessibility of care for patients with rare disorders like Pompe disease. Following is information about patient advocacy in general, and Pompe disease specifically, and why it is important for patients, caregivers and family members. What is Pompe disease? Pompe disease is a…

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bathdissertation, she analyzed Brexit’s long-term impact…